Support non-animal research to find a cure for ALSAlternatives to Animal Testing, Experimentation and Dissection - An Animal
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In an exciting medical breakthrough, researchers at
Johns Hopkins Medicine have transformed skin cells from patients with ALS
into brain cells that exhibit the disease characteristics. The cells,
donated by more than 20 ALS patients, were reprogrammed using genetic
engineering into induced pluripotent stem cells, and demonstrate the
pathology seen in ALS neural cells. Pluripotent stem cells have been
previously generated by scientists, but the Hopkins study is the first to
compile them into a “library” of human ALS cells, that will be shared
worldwide, enabling scientists to better study the disease.

Amyotrophic lateral sclerosis (ALS) is a debilitating, progressive
neurological disease that has no effective treatment or cure. ALS is often
referred to as Lou Gehrig’s disease, in memory of the star baseball player
whose life and career were cut in short in 1941. Tragically, in the decades
since Gehrig’s death, little progress has been made in treating ALS.

For reasons that are not fully understood, the disease causes nerve cells
in the brain and spinal cord to degenerate and die. Basic bodily functions,
like the ability to move, swallow and even breathe properly, deteriorate
over time. Total paralysis followed by death afflicts most patients in the
later stages of the disease. More than 30,000 people are diagnosed with ALS
in the US and 140,000 new cases are diagnosed each year worldwide.

In an exciting medical breakthrough, researchers at Johns Hopkins
Medicine have transformed skin cells from patients with ALS into brain cells
that exhibit the disease characteristics. The cells, donated by more than 20
ALS patients, were reprogrammed using genetic engineering into induced
pluripotent stem cells, and demonstrate the pathology seen in ALS neural
cells.

Pluripotent stem cells have been previously generated by scientists, but
the Hopkins study is the first to compile them into a “library” of human ALS
cells, that will be shared worldwide, enabling scientists to better study
the disease.

The study’s senior author, Dr. Jeffrey Rothstein, M.D., Ph.D, explains,
“We make brain cells out of the patient’s own skin. These human cellular
tools will serve as a platform to understand ALS and someday discover new
drugs to treat our patients.”

Since the 1990s, scientists and doctors have used mice to try to cure
ALS. Because ALS is a uniquely human disease, researchers have created mouse
“models” of ALS that try to incorporate some of the genetic defects found in
human ALS. Twenty-five years of research on mice has produced only a handful
of drugs that showed any efficacy in mice, but all failed when tried in
people.

Says Dr. Rothstein, “[A]fter 25 years, [mouse research] has not led to
the development of a drug that works in our patients. There has to be a sea
change in how we approach ALS.” Click here if you agree!

Last year, the ALS Association’s Ice Bucket Challenge garnered
international appeal and raised more than $220 million towards finding a
cure.

Given all we’ve learned about the failure of mouse research to produce
any successful treatments for ALS, let alone a cure, CAARE is calling upon
the ALS Association to put this money towards human research and modalities.
Click here to send a letter to ALSA in support of human-based research.

ALS patients deserve the best research towards developing a cure.
Resources must go towards productive human-relevant research rather than
being wasted on failed animal research. As Dr. Rothstein says, “Now we have
a real model for what’s wrong with my patients.”

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